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Gene Therapy and Drug Development

People with FOP have a difference in their genes that causes excess bone to grow. Gene therapy presents an exciting approach to treating FOP. While it holds great potential, gene therapy technology is currently limited.

We can silence, add, replace and, in some cases, edit genes. But we can’t do this for every cell in the body, all the time, for the life of the individual.

This is why traditional drug trials, like STOPFOP, are still needed.

Caring for People with FOP Today, Tomorrow and Into the Future

Your early funding gave us tools to explore which types of gene therapy — silencing (RNA), addition (DNA), or a combination of the two — have the highest potential to safely and effectively treat FOP.

The latest work of FOP researchers at the University of Massachusetts (UMASS) indicates that combination RNA/DNA treatment will be the most effective, with potentially the fewest side effects. However, RNA/DNA combination treatments require additional, time-consuming research that could add to the timeline between now and approval.
This is why UMASS and the IFOPA is looking at single RNA-delivered therapy in addition to combination RNA/DNA treatment. Single RNA treatment could be available sooner while RNA/DNA combination treatments are still in development.

There are other easy ways for you to learn more about gene therapy:

You’ll hear a lot about RNA interference and how it might lead to a treatment for FOP. Watch the video by TedEd — RNAi: Slicing, Dicing and Serving Your Cells by Alex Dainis to learn more about RNAi.

Watch the webinar featuring the IFOPA’s Research Director Danielle Kerkovich, PhD to learn more about gene therapy and how it might be used for FOP.

The American Society of Gene & Cell Therapy has created some wonderful tools to educate patients and families. Watch one or all of these videos from their Gene & Cell Therapy 101 educational series. The 2nd video, Viral Vectors Overview, will talk about AAV Vectors which are being studied at UMass to better understand their use in FOP.

By funding the STOPFOP clinical trial to test saracatinib, accelerating approval of RNA silencing and pursuing combination RNA/DNA treatment, the IFOPA is caring for those with FOP today, tomorrow and in the future. We won’t stop until there is a cure.

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